Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor-Reference-Cited by-同舟云学术

Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor

Published:2021-04-28 Issue:591 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Uchida Naoya¹²^ORCID,Ferrara Francesca³^ORCID,Drysdale Claire M.¹^ORCID,Yapundich Morgan¹^ORCID,Gamer Jackson¹^ORCID,Nassehi Tina¹^ORCID,DiNicola Julia¹^ORCID,Shibata Yoshitaka¹^ORCID,Wielgosz Matthew³^ORCID,Kim Yoon-Sang³^ORCID,Bauler Matthew⁴^ORCID,Throm Robert E.⁴^ORCID,Haro-Mora Juan J.¹^ORCID,Demirci Selami¹,Bonifacino Aylin C.⁵,Krouse Allen E.⁵,Linde N. Seth⁵^ORCID,Donahue Robert E.¹,Ryu Byoung³⁶,Tisdale John F.¹^ORCID

Affiliation:

1. Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.

2. Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan.

3. Department of Hematology, St. Jude Children’s Research Hospital (SJCRH), Memphis, TN 38105, USA.

4. Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.

5. Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.

6. Umoja Biopharma, 1920 Terry Ave., Seattle, WA 98101, USA.

Abstract

In xenograft mice and nonhuman primates, BCL11A interference and coexpression of a truncated erythropoietin receptor led to sustained hemoglobin F.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference54 articles.

1. Gene Therapy in a Patient with Sickle Cell Disease